Drug Discovery World

SEPTEMBER 14, 2023

Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format. Business updates Pfizer reported second quarter 2023 revenues totalling $12.7 billion, a decrease of $15.0 Vaccines were up 9.1%

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Drugs Vaccination Vaccine Medicine 59

WCG Clinical

MAY 28, 2024

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.

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Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment.

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Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1

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RNA In-Vivo Gene Therapy Genetic Disease 52

The Pharma Data

MARCH 8, 2021

Of 202 treated participants, no safety signals have been identified and of the 4 serious adverse events reported, none were considered to be study drug related. This program included assays such as Big Blue and PIG-a designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo.

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Trials In-Vivo Genotoxicity RNA 69

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

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pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

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Drug Discovery World

AUGUST 3, 2022

Emerging as a new treatment option in rare and orphan disease areas, oligonucleotide therapeutics have matured into a drug class with a broad indication spectrum. Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake.

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Bioavailability In-Vivo RNA In-Vitro 52

Delveinsight

FEBRUARY 25, 2021

Beam adds to drug delivery stable with USD 120 Million GuideTx buy. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs).

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DNA In-Vivo Genetics Medicine 52

Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT. The post RNA editing oligonucleotide selected for AATD treatment appeared first on Drug Discovery World (DDW).

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RNA In-Vivo Genotype Genetics 52

Drug Discovery World

SEPTEMBER 28, 2022

Ben Folwell, Principal Consultant at Citeline, maps out how AI, swarm learning and open access to clinical data could revolutionise drug discovery. . Its application in pharma, most commonly in drug discovery, has been steadily growing with global partnerships between tech vendors and pharma companies becoming more commonplace.

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Pharma Companies Drugs In-Vivo Genetics 52

Pharmaceutical Technology

AUGUST 21, 2022

The China National Medical Products Administration (NMPA) has granted clearance for Ascletis Pharma’s Investigational New Drug (IND) application of its drug candidate, ASC10, for Covid-19. An RNA-dependent RNA polymerase (RdRp) inhibitor, ASC10 is an oral double prodrug. D-N4-hydroxycytidine (NHC) in vivo.

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RNA In-Vivo Clinical Development Drugs 162

Drug Discovery World

SEPTEMBER 6, 2022

There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. Among these include Zolgensma, Novartis’ gene therapy drug for spinal muscular atrophy (SMA), which made headlines in the UK for its £1.79 billion, compared to $19.9 million price point per dose. Cancer is king .

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Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

APRIL 12, 2023

This paid-for advertorial by CN Bio appeared in DDW Volume 24 – Issue 2, Spring 2023 Despite advancements, the drug development process remains inefficient. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

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Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

JUNE 17, 2024

One dose of Nutcracker Therapeutics’ NTX-472 in vivo rapidly depleted B cells with no detectable binding to red blood cells. We’re proud to be one of the first RNA therapeutics companies to engineer a multispecific antibody,” said Chief Scientific Officer Samuel Deutsch. “We

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Antibody In-Vivo In-Vitro Engineer 59

Drug Discovery World

JANUARY 20, 2023

This paid-for advertorial by CN Bio appeared in the SLAS 2023 Supplement, Volume 24 – Issue 1, Winter 2022/2023 Despite advancements, the drug development process remains inefficient. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

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Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

SEPTEMBER 25, 2023

RNA therapeutics company SiSaf has published positive safety and efficacy data for its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). Co-author on the paper, Dr Suzanne Saffie-Siebert, CEO of SiSaf, said: “Targeting the bone safely and effectively is a challenge for RNA-based therapies.

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RNA In-Vivo Clinical Trials Clinical Trials 59

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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Gene Editing Trials Drugs Gene 98

Drug Discovery World

OCTOBER 13, 2022

Prior to the pandemic, messenger RNA (mRNA) drug development was niche and predominantly pointed at different disease targets, including infectious diseases and cancers. One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. Why mRNA has potential .

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Drugs Manufacturing Vaccination Vaccine 52

Drug Discovery World

NOVEMBER 15, 2022

Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. . It has made its way into almost every aspect of drug discovery, whether it’s exploring basic biology, modelling diseases, identifying and validating targets, or performing drug screens. .

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DNA Drugs Antibody Engineer 98

The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

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Vaccination Vaccine Immune Response Protein 52

Drug Discovery World

JANUARY 17, 2024

Challenges in the Oncology Clinical Trials Space The oncology market is a key growth driver for the pharma industry, with global drug revenues expected to reach US$390 billion by 2027 and the annual number of new cancer cases set to grow from 18 to 27 million by 2040 1,2.

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Medicine DNA Genome Genomics 59

Delveinsight

JANUARY 18, 2021

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. GeneTx and Ultragenyx are co-developing the therapy.

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Marketing Gene Drugs Protein 52

XTalks

APRIL 22, 2022

Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. Among these is the receptor occupancy assay, which is a powerful method used to understand how a therapeutic drug binds to (or occupies ) its receptors on the surface of certain cells.

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Clinical Trials Clinical Trials Trials Antibody 98

Drug Discovery World

JULY 18, 2023

Incorporating in vivo pooled CRISPR screens has allowed for the interrogation of thousands of genetic perturbations in cells, leading to the determination of the involvement of molecules (PTPN2, ADAR1, or DHX37) in immune checkpoint inhibitor (ICI) responsiveness and T cell activation.

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DNA Genome Genomics RNA 98

Drug Discovery World

DECEMBER 15, 2022

Prime editing, while also employing a single strand nick, expands this scope to all 12 possible changes using a more complex prime editing guide RNA (pegRNA). Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo.

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Gene Editing Gene In-Vivo Genome 52

Roots Analysis

AUGUST 31, 2023

It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E.

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Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. Lightcast is developing a novel, programmable microfluidic platform that allows precise and highly flexible control of individual microdroplets using software-generated light patterns.

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Antibody Protein Engineer In-Vivo 59

Drug Discovery World

NOVEMBER 13, 2023

Noel Pauli, PhD, Group Leader, Antibody Engineering, Adimab, on: ‘Harnessing in vivo diversities with a yeast-based platform for the discovery of antibodies against multi-pass membrane proteins’. Oliver Hartley, PhD, Vice President, Drug Discovery, Orion Biotechnology, on: ‘Targeting small protein GPCRs by engineering their natural ligands’.

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Protein Antibody Engineer Scientist 59

Drug Discovery World

JANUARY 23, 2023

This circumvents several technical challenges inherent to the genetic manipulation of differentiated cells in ex vivo culture or intact tissues. In cell therapy, regenerative and adoptive immunotherapies continue to bridge the gap between genetic engineering strategies and their clinical application.

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Gene Editing Gene Engineer DNA 52

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. to £104.7m

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Drugs Gene Clinical Trials Clinical Trials 59

Drug Discovery World

OCTOBER 20, 2022

It has been used to investigate rare cell populations, screen potential drug therapeutics, and examine sample phenotypes for diagnostic applications 2. Fibroblasts in the tumour microenvironment. Cancer associated fibroblasts (CAFs) have recently emerged as key players in the tumour microenvironment. Conventional flow cytometry.

Antibody 52

Antibody In-Vivo Life Science Development 52

Drug Discovery World

MAY 10, 2023

Dr Megan MacBride and Dr Caroline Horizny Mitchell , Taconic, examine recent changes to animal testing in drug design. Drug discovery and development is an arduous process that can cost upwards of $2.6 In August 2022, the ICH issued an addendum to its S1B guidelines that allows certain drugs to forego two-year rat studies 1.

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Bioassay Genotoxicity Drugs In-Vivo 52